NCT01230021已完成3 期
A Phase 3b Trial Investigating the Pharmacokinetics and Safety Profile of a Single Intravenous Dose of rFXIII in Paediatric (1 to Less Than 6 Years Old) Subjects With Congenital FXIII A-subunit Deficiency
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Area Under the Concentration vs. Time Curve (AUC)
研究概览
简要总结
This trial is conducted in Europe and United States of America (USA). The aim of this clinical trial is to investigate the pharmacokinetics (at which rate the substance is distributed and eliminated from the body) and the safety profile of catridecacog (recombinant factor XIII (rFXIII)) in children with congenital FXIII A-subunit deficiency. Young children (1 to less than 6 years old) with congenital FXIII deficiency are evaluated.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 6 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed Informed Consent by subject's parents or subject's legally acceptable representative before any trial related activities. Trial related activities are any procedures that would not have been performed during the normal management of the subject
- •Age 1 to less than 6 years old at the time of enrolment
- •Congenital FXIII subunit-A deficiency previously documented by genotyping or evaluated by genotyping through blood sampling at screening visit
- •Body weight at least 10 kg
排除标准
- •Known antibodies to FXIII
- •Hereditary or acquired coagulation disorder other than FXIII A-subunit congenital deficiency
- •Platelet count (thrombocytes) of less than 50 × 10^9/L (at screening visit)
- •Previous history of autoimmune disorder involving autoantibodies e.g., systemic lupus erythematosus
- •Previous history of arterial or venous thromboembolic events e.g., cerebrovascular accident or deep vein thrombosis
- •Known or suspected allergy to trial product or related products
- •Any surgical procedure in the 30 days prior to enrolment and any planned surgery during the trial period
- •Any disease or condition which, judged by the Investigator, could imply a potential hazard to the subject or interfere with the trial participation or trial outcome including renal and/or liver dysfunction
研究组 & 干预措施
recombinant factor XIII
Experimental
干预措施: catridecacog (Drug)
结局指标
主要结局
Area Under the Concentration vs. Time Curve (AUC)
时间窗: At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing
A measure of the exposure. Blood samples for the PK assessment were drawn pre-dose and up to 30 days after dosing. The PK of FXIII in children was assessed after a single i.v. dose of rFXIII 35 IU/kg.
次要结局
- Area Under the Concentration vs. Time Curve (AUC0-∞)(From day 0 to day 30)
- Maximum Plasma Concentration (Cmax) for FXIII(At pre-dose, 30 minutes, 24 hours, 7, 14, 21 and 30 days after dosing)
- Terminal Half-life (t½)(From day 0 to day 30)
- Mean Residence Time (MRT)(From day 0 to day 30)
- Coagulation Related Parameters - Activated Partial Thromboplastin Time (aPTT, Seconds)(Day 0 and day 30)
- Coagulation Related Parameters - Prothrombin Time (PT) (Seconds)(Day 0 and day 30)
- Total Plasma Clearance (CL)(From day 0 to day 30)
- Volume of Distribution at Steady State (Vss)(At steady state)
- Percentage of Subjects With One or More Adverse Events (AEs) Recorded(From day 0 to day 30)
- Percentage of Subjects With One or More Serious Adverse Events (SAEs)(From day 0 to day 30)
- Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors (Neutralising Antibodies Against Factor XIII)(At screening and day 30)
- Coagulation Related Parameters - Fibrinogen(Day 0 and at day 30)
- Clot Solubility Test (Evaluated as Normal/Abnormal)(Day 0 and day 30)
- Vital Signs - Pulse(Day 0 and day 30)
- Vital Signs - Blood Pressure (Systolic and Diastolic)(Day 0 and day 30)
- Physical Examination (Evaluated as Normal/Abnormal)(From day 0 to day 30)
研究者
研究点 (1)
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